A phase 1 trial of a human thymus-expressed apoptosis factor in patients with advanced solid tumors: Interim report.

e14018 Background: This apoptosis factor (NEROFE) is a 14-a.a. modified form of a hormone-like peptide present in the human thymus, which plays a key role in immune system regulation. In preclinical evaluation, it induced apoptosis in cancer cells, inhibited angiogenesis/metastasis, and showed in vivo efficacy with no toxicity. Methods: This ongoing, single-center, first-in-human, 3+3 dose-escalation study of this factor given I.V. at 6 mg/m2-96 mg/m2over 5 3-pt cohorts (3 times a week; 28-day cycle) examines the MTD, safety, PK profile, and anti-tumor activity in pts with advanced solid tumors. Pts undergo tumor assessments every other cycle. Samples for PK are taken on days 1 of cycles 1 and 2. Results: To date, 14 previously treated (57% ≥ 3 lines) and 1 first line pts (M = 8, F = 7, median age: 63) with advanced/metastatic solid tumors have been enrolled. MTD has not been reached. Treatment was well-tolerated with no cumulative toxicity. The most frequently observed AEs (G < 2) in the first 4 cohorts ...